Category REGENERATIVE MEDICINE

Monash University study uncovers a new understanding of how mesenchymal stromal cells benefit patients in cell therapy

The therapeutic benefit to patients receiving mesenchymal stromal cell (MSC) therapy is not because the injected cells remain viable, but because of cell death, researchers at the Monash Biomedicine Discovery Institute (BDI) have found. In recent years, significant efforts have been made to develop stem cell-based therapies for difficult-to-treat diseases. MSC therapy is regenerative cell-based

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Cell therapies, Gene therapy and CAR-T cells highlighted in EFPIA Pipeline Review 2021 Update

The new EFPIA Pipeline Review reveals that infectious, inflammatory, cancer, Alzheimer’s and rare disesases areas are among the top priorities of researchers running clinical trials today to investigate new medicines and vaccines. The comprehensive report found that in 2020 alone, around 5,000 clinical trials were launched – despite the disruption caused by the COVID-19 pandemic. In fact, the volume of trials has

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Ernst & Young white paper details how the Cell Gene Therapy industry can collaborate to advance the field

Harnessing the power of cell and gene therapies will require a next-generation level of collaboration not seen before the COVID-19 pandemic. In brief Before cell and gene therapies can meet increasing demand at the necessary speed and scale, the industry must address key challenges. A fractured supply chain, complicated manufacturing and unprecedented pricing are among

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The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study published in Blood

In the last issue of Blood, Boulos et al analyze the cost-effectiveness of factor IX gene therapy in patients with severe hemophilia B, in a microsimulation Markov model. Hemophilia is an X-linked disease that affects 400000 people worldwide, of whom 15% have hemophilia B caused by deficiency of factor IX (FIX), which affected the offspring of Queen Victoria,

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SP-101, an investigational inhaled gene therapy for cystic fibrosis, shows potential in animal study

Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD, Spirovant’s vice president of research, presented the data at the 2021 North American Cystic Fibrosis Conference (NACFC). Her

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Children Hospital of Philadelphia Researchers Develop a New Class of CAR-T Cells that Target Previously Untargetable Cancer Drivers

In a breakthrough for the treatment of aggressive solid cancers, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach. Using the power of large data sets and advanced computational approaches,

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A review on Mesenchymal stem cells-derived exosomes for drug delivery

Mesenchymal stem cells (MSCs) are multi-functional stem cells that are present in multiple human tissues and can be found in the spinal cord, umbilical cord blood, umbilical cord tissue, placenta tissue, adipose tissue, etc.. With low immunogenicity, multi-directional diferentiation ability, in particular homing ability, MSCs have signifcant research potential in cardiovascular diseases, nervous diseases, and

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Stem cell treatment failed to reduce inflammation in Multiple Sclerosis

Treatment with mesenchymal stem cells (MSCs) has failed to significantly reduce inflammation in the brain of adults with multiple sclerosis (MS) in a phase 2 clinical trial. The into-the-vein treatment also failed to improve other clinical aspects of the condition. The results were shared in a study titled “Safety, tolerability, and activity of mesenchymal stem cells versus placebo

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Gene Therapies against Rare diseases Boosted by NIH, FDA and Industry Alliance

The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke

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